World-first CRISPR therapy could ‘transform’ treatment for rare genetic diseases, but key challenges lie ahead
It has been described as a revolutionary technology — and won its inventors a Nobel Prize.CRISPR gene editing, often simply...
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Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment
News ReleaseThursday, May 15, 2025 NIH-supported gene-editing platform lays groundwork to rapidly develop treatments for other rare genetic diseases.A research...
Dallas scientists granted $420K to test gene therapy for rare neurological disease
Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by age...
The Future of Gene-Editing Treatments for Rare Diseases < Yale School of Medicine
Angelman syndrome is a rare genetic disorder caused by a mutation on chromosome 15, which hinders the production of a...
Gene therapy shows promise for treating rare muscle disease
A University of Alberta research team has taken the first step toward proving its innovative gene therapy can successfully treat...
ASH: Gene therapy for rare genetic disorder, molecular insights into leukemia growth and more
Researchers from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA will present their...
Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord
Introduction Classically, the majority of medical treatments have been developed for diseases affecting large number of patients and patients with...
Gene Therapy Promising for Rare Genetic Cardiomyopathy
CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or stabilize the...
Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate
Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not...
